ROTTERDAM, Netherlands--(BUSINESS WIRE)--Treeway, a biotech company developing therapies against amyotrophic lateral sclerosis (ALS), today announced the completion of its Phase I trial of lead program TW001, an oral formulation of edaravone. The study compared the bioavailability of TW001 in its novel market formulation to Radicava®, an FDA approved, intravenous formulation of edaravone, indicated for the treatment of ALS. The study indicates increased bioavailability of TW001 in a single-dose treatment whilst no safety concerns developed within the subject group, implying the potential of the novel formulation to improve treatment strategies for ALS patients. Based on these results, Treeway has initiated the upscaling program and preparations for a pivotal Phase 3 study. Edaravone is a neuroprotective agent that reduces oxidative stress, a key contributor to neuronal death in ALS.
“It is Treeway’s mission to identify novel treatment routes for ALS patients and we believe that this study indicates that an oral formulation of edavarone may offer patients a better quality-of-life through a simpler and more effective route of administration,” said Ronald van der Geest, Chief Development Officer of Treeway. “Given the nature of this program and based on the positive results we have seen in this comparative study, we can initiate a pivotal Phase 3 study and move rapidly toward bringing TW001 to patients.”
The randomized, cross-over comparative bioavailability trial tested a single oral dose of 140 mg TW001, compared to a 1-hour intravenous infusion of 60 mg edaravone in 18 healthy subjects. In this study, the bioavailability of the novel oral formulation exceeded the bioavailability of the intravenous infusion of Radicava®. Within the subject group of the study the treatment was well-tolerated and no safety concerns arose during the conduct of the study. Furthermore, ongoing formulation studies also suggest that the formulation is stable.
Merit Cudkowicz, MD, Chief Neurology Service and Director of the ALS Clinic at the Massachusetts General Hospital, a key advisor to the company, commented:
“The results of this Phase I study are a promising sign for ALS patients, who are in high need of better and more patient-friendly treatments for their disease. I regard this study to be a key milestone for the development of TW001 in ALS and I look forward to supporting the company’s scientific and clinical progress.”
TW001, Treeway’s lead compound, is a proprietary oral formulation of the FDA-approved intravenous formulation of edaravone (Radicava®). In clinical studies, edavarone was shown to slow down disease progression in ALS patients by one-third through its neuroprotective properties. TW001 is developed as a disease modifying drug capable of treating all phenotypes of ALS, for daily use to improve patient convenience.
This press release is intended to communicate the scientific outcomes of the Phase 1 trial of TW001. TW001 is an investigational new drug and is not available for commercial distribution. The outcomes of the Phase 1 study will have to be confirmed by an additional pivotal Phase 3 study and subsequent registration of the product before any definitive conclusions or claims regarding the safety and effectiveness of TW001 can be made.
Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s Disease, is a progressive neurodegenerative rare disease that causes muscle weakness, disability and eventually death. The average survival from diagnosis to death is three to four years. The disease most frequently occurs between the age of forty to sixty. In Europe and the United States, the disease affects about two people per 100,000 per year. Currently, there are about 400,000 patients with ALS in the world. To date, there is no cure for ALS.
Treeway is a privately-owned biotechnology company, focused on the development of novel improved therapies for ALS. It was founded in 2012. The management team has wide expertise in drug development and is strongly embedded in the patient, physician and academic community, through a range of partnerships and collaborations. Treeway’s development programs are co-funded by the United States Department of Defense (DoD) and The Dutch Ministry of Economic Affairs’ Innovation Credit.